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Novel targets for Huntington’s disease: future prospects

Authors Mason S, Barker R

Received 24 December 2015

Accepted for publication 9 February 2016

Published 4 May 2016 Volume 2016:6 Pages 25—36


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Prof. Dr. Thomas Müller

Sarah L Mason,1 Roger A Barker1,2

1John van Geest Centre for Brain Repair, 2Department of Clinical Neuroscience, University of Cambridge, Cambridge, UK

Huntington’s disease (HD) is an incurable, inherited, progressive, neurodegenerative disorder that is characterized by a triad of motor, cognitive, and psychiatric problems. Despite the noticeable increase in therapeutic trials in HD in the last 20 years, there have, to date, been very few significant advances. The main hope for new and emerging therapeutics for HD is to develop a neuroprotective compound capable of slowing down or even stopping the progression of the disease and ultimately prevent the subtle early signs from developing into manifest disease. Recently, there has been a noticeable shift away from symptomatic therapies in favor of more mechanistic-based interventions, a change driven by a better understanding of the pathogenesis of this disorder. In this review, we discuss the status of, and supporting evidence for, potential novel treatments of HD that are currently under development or have reached the level of early Phase I/II clinical trials.

Keywords: disease modification, transcript dysregulation, glial modulation, cell death

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